日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Modelling POLG mutations in mice unravels a critical role of POLγΒ in regulating phenotypic severity.

在小鼠中模拟 POLG 突变揭示了 POLγÎ' 在调节表型严重程度中的关键作用

Corrà Samantha, Zuppardo Alessandro, Valenzuela Sebastian, Jenninger Louise, Cerutti Raffaele, Sillamaa Sirelin, Hoberg Emily, Johansson Katarina A S, Rovsnik Urska, Volta Sara, Silva-Pinheiro Pedro, Davis Hannah, Trifunovic Aleksandra, Minczuk Michal, Gustafsson Claes M, Suomalainen Anu, Zeviani Massimo, Macao Bertil, Zhu Xuefeng, Falkenberg Maria, Viscomi Carlo

Clinically translatable mitochondrial gene therapy in muscle using tandem mtZFN architecture.

利用串联 mtZFN 结构在肌肉中进行临床转化线粒体基因治疗

Nash Pavel A, Turner Keira M, Powell Christopher A, Van Haute Lindsey, Silva-Pinheiro Pedro, Bubeck Felix, Wiedtke Ellen, Marques Eloïse, Ryan Dylan G, Grimm Dirk, Gammage Payam A, Minczuk Michal

Correction to 'DNA polymerase gamma mutations that impair holoenzyme stability cause catalytic subunit depletion'

更正“损害全酶稳定性的DNA聚合酶γ突变导致催化亚基耗竭”

Silva-Pinheiro, Pedro; Pardo-Hernández, Carlos; Reyes, Aurelio; Tilokani, Lisa; Mishra, Anup; Cerutti, Raffaele; Li, Shuaifeng; Rozsivalova, Dieu-Hien; Valenzuela, Sebastian; Dogan, Sukru A; Peter, Bradley; Fernández-Silva, Patricio; Trifunovic, Aleksandra; Prudent, Julien; Minczuk, Michal; Bindoff, Laurence; Macao, Bertil; Zeviani, Massimo; Falkenberg, Maria; Viscomi, Carlo